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NCT04189484

Pharmacodynamic Biomarkers to Support Biosimilar Development: PCSK9 Inhibitors

Completed Phase 1 Results posted Last updated 22 April 2024
What this trial tests

Phase 1 trial testing Evolocumab in Healthy Subjects in 72 participants. Completed in 7 April 2021.

Timeline
7 January 2020
Primary endpoint
7 April 2021
7 April 2021

Quick facts

Lead sponsorFood and Drug Administration (FDA)
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingdouble
Primary purposeother
Enrollment72
Start date7 January 2020
Primary completion7 April 2021
Estimated completion7 April 2021
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Food and Drug Administration (FDA) — full company profile →

Who can join

Adults 18 to 55, any sex, with Healthy Subjects or Pharmacokinetics. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Baseline-adjusted Area Under Effect Curve (AUEC) for LDL-C for Evolocumab and Alirocumab Primary · Day -1 and 0h (pre-dose), 24h (post-dose); once on Day 2, 3, 4, 5, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

The values and variability of AUEC for LDL-C at low, intermediate-low, intermediate-high, and high doses of evolocumab and alirocumab. AUEC was calculated as the baseline-subtracted area under the LDL-C concentration-time curve expressed in units of mg/(dL\*day). More negative AUEC values represent greater reductions in LDL-C exposure from baseline. The standard deviation is noted in parentheses. For each subject and dose, the maximum decrease from baseline LDL-C was determined using all sampled timepoints. AUEC values were log-transformed and an ANCOVA model was used to calculate geometric me

GroupValue95% CI
Arm A: Evolocumab Low Dose-380± 228
Arm B: Evolocumab Intermediate Low Dose-754± 573
Arm C: Evolocumab Intermediate High Dose-866± 658
Arm D: Evolocumab High Dose-1832± 806
Arm E: Alirocumab Low Dose-580± 360
Arm F: Alirocumab Intermediate Low Dose-581± 319
Arm G: Alirocumab Intermediate High Dose-668± 828
Arm H: Alirocumab High Dose-1902± 361
Arm I: Placebo-350± 881
Maximum Change From Baseline for LDL-C for Evolocumab and Alirocumab Primary · Day -1 and 0h (pre-dose), 24h (post-dose); once on Day 2, 3, 4, 5, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

The values and variability of maximum change from baseline for LDL-C at low, intermediate low, intermediate high, and high doses of evolocumab and alirocumab

GroupValue95% CI
Arm A: Evolocumab Low Dose-27.7± 11.8
Arm B: Evolocumab Intermediate Low Dose-45.1± 26.9
Arm C: Evolocumab Intermediate High Dose-49.9± 26.2
Arm D: Evolocumab High Dose-70.1± 25.0
Arm E: Alirocumab Low Dose-35.8± 16.3
Arm F: Alirocumab Intermediate Low Dose-40.6± 8.1
Arm G: Alirocumab Intermediate High Dose-47.9± 18.8
Arm H: Alirocumab High Dose-54.6± 12.1
Arm I: Placebo-30.1± 18.7
Baseline-adjusted AUEC for Apolipoprotein B (ApoB) for Evolocumab and Alirocumab Secondary · Day -1 and 0h (pre-dose), 24h (post-dose); once on Day 2, 3, 4, 5, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

The values and variability of AUEC for ApoB at low, intermediate-low, intermediate-high, and high doses of evolocumab and alirocumab. AUEC was calculated as the baseline-subtracted area under the ApoB concentration-time curve expressed in units of mg/(dL\*day). More negative AUEC values represent greater reductions in ApoB exposure from baseline. The standard deviation is noted in parentheses. For each subject and dose, the maximum decrease from baseline ApoB was determined using all sampled timepoints. AUEC values were log-transformed and an ANCOVA model was used to calculate geometric means

GroupValue95% CI
Arm A: Evolocumab Low Dose-207± 313
Arm B: Evolocumab Intermediate Low Dose-482± 434
Arm C: Evolocumab Intermediate High Dose-676± 453
Arm D: Evolocumab High Dose-1094± 952
Arm E: Alirocumab Low Dose-469± 290
Arm F: Alirocumab Intermediate Low Dose-462± 231
Arm G: Alirocumab Intermediate High Dose-128± 536
Arm H: Alirocumab High Dose-1087± 707
Arm I: Placebo-428± 560
Maximum Change From Baseline for apoB for Evolocumab and Alirocumab Secondary · Day -1 and 0h (pre-dose), 24h (post-dose); once on Day 2, 3, 4, 5, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

The values and variability of maximal difference at a single time-point for ApoB at low, intermediate low, intermediate high, and high doses of evolocumab and alirocumab

GroupValue95% CI
Arm A: Evolocumab Low Dose24± 12
Arm B: Evolocumab Intermediate Low Dose32± 17
Arm C: Evolocumab Intermediate High Dose36± 15
Arm D: Evolocumab High Dose46± 16
Arm E: Alirocumab Low Dose23± 9
Arm F: Alirocumab Intermediate Low Dose29± 6
Arm G: Alirocumab Intermediate High Dose28± 8
Arm H: Alirocumab High Dose37± 7
Arm I: Placebo17± 7
Maximum Concentration (Cmax) for Evolocumab and Alirocumab Secondary · 0 (pre-dose), 0.5, 1, 2, 3, 4, 6, 8,12, 24, hours post-dose; once on Day 2, 3, 4, 5, 6, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

The values and variability of Cmax at low, intermediate low, intermediate high, and high doses of evolocumab and alirocumab

GroupValue95% CI
Arm A: Evolocumab Low Dose0.7± 28
Arm B: Evolocumab Intermediate Low Dose0.5± 62
Arm C: Evolocumab Intermediate High Dose2.0± 54
Arm D: Evolocumab High Dose6.2± 36
Arm E: Alirocumab Low Dose0.8± 27
Arm F: Alirocumab Intermediate Low Dose1.7± 73
Arm G: Alirocumab Intermediate High Dose2.9± 61
Arm H: Alirocumab High Dose6.9± 41
Area Under the Curve (AUC) for Evolocumab and Alirocumab Secondary · 0 (pre-dose), 0.5, 1, 2, 3, 4, 6, 8,12, 24, hours post-dose; once on Day 2, 3, 4, 5, 6, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

The values and variability of AUC at low, intermediate low, intermediate high, and high doses of evolocumab and alirocumab

GroupValue95% CI
Arm A: Evolocumab Low Dose2.4± 67
Arm B: Evolocumab Intermediate Low Dose0.8± 91
Arm C: Evolocumab Intermediate High Dose12.9± 71
Arm D: Evolocumab High Dose65.7± 57
Arm E: Alirocumab Low Dose8.3± 55
Arm F: Alirocumab Intermediate Low Dose17.1± 57
Arm G: Alirocumab Intermediate High Dose38.5± 92
Arm H: Alirocumab High Dose107.9± 58
Dose-response Parameters (Slope) for LDL-C Area Under the Effect Curve Models for Evolocumab and Alirocumab Secondary · Day -1 and 0h (pre-dose), 24h (post-dose); once on Day 2, 3, 4, 5, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

Model parameter (slope) for LDL-C area under the effect curve model: Mean slope value calculated after combining data from low, intermediate low, intermediate high, and high doses of evolocumab or alirocumab with placebo data. The units of measure for slope are mg/dL•day / (mg dose).

GroupValue95% CI
Evolocumab: Area Under the Effect Curve Model for LDL-C-11-15 – -6
Alirocumab: Area Under the Effect Curve Model for LDL-C-15-21 – -9
Pharmacodynamic Model Parameter, Maximum Effect (Emax), for LDL-C Maximum Change From Baseline Models With Evolocumab and Alirocumab Secondary · Day -1 and 0h (pre-dose), 24h (post-dose); once on Day 2, 3, 4, 5, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

Model parameter (Emax) for LDL-C maximum change from baseline curve models calculated after combining data from low, intermediate low, intermediate high, and high doses of evolocumab or alirocumab with placebo data.

GroupValue95% CI
Evolocumab: Maximum Change From Baseline Model for LDL-C8455 – 113
Alirocumab: Maximum Change From Baseline Model for LDL-C6250 – 75
Pharmacodynamic Model Parameter, ED50 (Half Maximal Effect Dose), for LDL-C Maximum Change From Baseline Models With Evolocumab and Alirocumab Secondary · Day -1 and 0h (pre-dose), 24h (post-dose); once on Day 2, 3, 4, 5, 7, 10, 14, 21, 28, 35, and 42 for all Arms. Additionally, once on Day 56 for Arms C, D, G, H, & I; and once on Day 70 and 84 for Arms D, H, & I.

Model parameter (ED50) for LDL-C maximum change from baseline curve models calculated after combining data from low, intermediate low, intermediate high, and high doses of evolocumab or alirocumab with placebo data.

GroupValue95% CI
Evolocumab: Maximum Change From Baseline Model for LDL-C320 – 66
Alirocumab: Maximum Change From Baseline Model for LDL-C145 – 23

Adverse events — posted to ClinicalTrials.gov

Time frame: 42 days for subjects in treatment groups A, B, E, and F; 56 days for subjects in treatment groups C and G; and 84 days for subjects in treatment groups D, H, and I. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Arm A: Evolocumab Low Dose
Serious: 0/8 (0%)
Deaths: 0/8
Arm B: Evolocumab Intermediate Low Dose
Serious: 0/8 (0%)
Deaths: 0/8
Arm C: Evolocumab Intermediate High Dose
Serious: 0/8 (0%)
Deaths: 0/8
Arm D: Evolocumab High Dose
Serious: 0/8 (0%)
Deaths: 0/8
Arm E: Alirocumab Low Dose
Serious: 0/8 (0%)
Deaths: 0/8
Arm F: Alirocumab Intermediate Low Dose
Serious: 0/8 (0%)
Deaths: 0/8
Arm G: Alirocumab Intermediate High Dose
Serious: 0/8 (0%)
Deaths: 0/8
Arm H: Alirocumab High Dose
Serious: 0/8 (0%)
Deaths: 0/8
Arm I: Placebo
Serious: 0/8 (0%)
Deaths: 0/8
Other adverse events (34 terms — click to expand)

ReactionSystemArm A: Evolocumab Low DoseArm B: Evolocumab Intermed…Arm C: Evolocumab Intermed…Arm D: Evolocumab High DoseArm E: Alirocumab Low DoseArm F: Alirocumab Intermed…Arm G: Alirocumab Intermed…Arm H: Alirocumab High DoseArm I: Placebo
Vessel puncture site painGeneral disorders
HeadacheNervous system disorders
Back painMusculoskeletal and connective tissue disorders
MyalgiaMusculoskeletal and connective tissue disorders
CoughRespiratory, thoracic and mediastinal disorders
Nasal congestionRespiratory, thoracic and mediastinal disorders
RhinorrheaRespiratory, thoracic and mediastinal disorders
Corneal abrasionEye disorders
Eye irritationEye disorders
Abdominal discomfortGastrointestinal disorders
ConstipationGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
Dry mouthGastrointestinal disorders
FlatulenceGastrointestinal disorders
NauseaGastrointestinal disorders
VomitingGastrointestinal disorders
Feeling hotGeneral disorders
Injection site painGeneral disorders
PyrexiaGeneral disorders
Vessel puncture site haemorrhageGeneral disorders
Anal abscessInfections and infestations
Bronchitis bacterialInfections and infestations
ArthralgiaMusculoskeletal and connective tissue disorders
Limb discomfortMusculoskeletal and connective tissue disorders
Muscle spasmsMusculoskeletal and connective tissue disorders
DysgeusiaNervous system disorders
Head discomfortNervous system disorders
DysphoniaRespiratory, thoracic and mediastinal disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
PharyngitisRespiratory, thoracic and mediastinal disorders
Sneezing excessiveRespiratory, thoracic and mediastinal disorders
TonsillolithRespiratory, thoracic and mediastinal disorders
WheezingRespiratory, thoracic and mediastinal disorders
Skin abrasionSkin and subcutaneous tissue disorders

Data from ClinicalTrials.gov NCT04189484 adverse events section.

Sponsor's own description

This study is designed to assess pharmacokinetics and pharmacodynamics of evolocumab and alirocumab across an appropriate dose range to inform clinical trial operating characteristics for future clinical pharmacology pharmacodynamics similarity studies. This is a randomized, placebo-controlled, single-dose, parallel arm study in 72 healthy subjects assigned to one of four dose groups (low, intermediate low, intermediate high, and high) of each drug (evolocumab and alirocumab ) or placebo.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04189484.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing